Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
APR-246+ azacitidine · 1 trial · 1 indication
To compare the complete response rate, defined as the proportion of patients who achieve complete remission (CR) with APR 246 + azacitidine treatment vs. azacitidine only.
| Arm | Type | Description |
|---|---|---|
| Experimental arm: APR-246 + azacitidine | EXPERIMENTAL | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): |
| Control arm: Azacitidine | EXPERIMENTAL | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): |
| Name | Type | Description |
|---|---|---|
| APR-246 + azacitidine | DRUG | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine |
| Azacitidine | DRUG | Patients will be randomized (1:1) to one of two arms: stratified by age (\< 65 years versus ≥ 65): Experimental arm: APR-246 + azacitidine; or Control arm: Azacitidine |
Inclusion Criteria: * Signed Informed Consent (ICF) and is able to comply with protocol requirements * Documented diagnosis of MDS, according to World Health Organization (WHO) classification * Patient has adequate organ function as defined by the following laboratory values: 1. Creatinine clear...
APR-246 is an investigational small molecule being studied for the treatment of several cancers, including myelodysplastic syndromes (MDS), acute myeloid leukemia, high-grade serous ovarian cancer, and bladder cancer. It is also being evaluated in hematologic neoplasms and myeloid malignancies. The drug is not yet approved and remains in clinical development.
APR-246 is designed to reactivate the tumor suppressor protein p53, which is often mutated in cancer cells. By restoring p53 function, the drug aims to induce apoptosis, or programmed cell death, in malignant cells. This mechanism is being studied across multiple cancer types, particularly those with TP53 mutations.
APR-246 is being developed by Aprea Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol APRE. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various oncology indications.
APR-246 has completed Phase 1, Phase 2, and Phase 3 clinical trials. The most advanced study, NCT03745716, was a Phase 3 trial in patients with TP53 mutant myelodysplastic syndromes. However, the drug remains investigational and has not received FDA approval.
APR-246 has been studied in four completed clinical trials. NCT00900614 was a Phase 1 safety study in refractory hematologic or prostate cancer. NCT03268382 was a Phase 2 trial in platinum-resistant ovarian cancer. NCT03745716 was a Phase 3 trial in TP53 mutant MDS, and NCT03931291 was a Phase 2 trial in AML or MDS after stem cell transplant.
Yes, APR-246 is also known as eprenetapopt. This alternative name is used in some clinical and scientific literature. Both names refer to the same investigational small molecule being developed by Aprea Therapeutics for the treatment of p53-mutant cancers.